Am­i­cus gets hit with an 'un­ex­pect­ed' set­back on a gene ther­a­py tapped to lead $600M SPAC spin­out

Am­i­cus has tak­en the axe and chopped a gene ther­a­py for Bat­ten dis­ease out of the pipeline af­ter an “un­ex­pect­ed” set­back in the clin­ic.

In a cor­po­rate up­date on Mon­day, the biotech re­port­ed that its long-term ex­ten­sion da­ta from a Phase I/II study re­vealed that the ini­tial sta­bi­liza­tion they had achieved in pa­tients dur­ing the first 2 years wasn’t sus­tain­able. So re­searchers are drop­ping the CLN6 Bat­ten dis­ease gene ther­a­py pro­gram and turn­ing to a CLN3 pro­gram, al­so for Bat­ten dis­ease, with a “high­er dose, dif­fer­ent pro­mot­er, and in­tra-cis­ter­na magna route of de­liv­ery.”

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